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New treatment option for Generalized Myasthenia Gravis
Official title A Study of Efgartigimod PH20 SC in Children Between 2 and Less Than 18 Years of Age With Generalized Myasthenia Gravis
ClinicalTrials.gov ID: NCT06392386
What this study is testing
What is Efgartigimod PH20 SC?
Efgartigimod PH20 SC is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for generalized myasthenia gravis.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The purpose of this study is to measure the pharmacokinetics (PK), pharmacodynamics (PD), safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to \<18 years. The primary goal is to confirm an appropriate dose of efgartigimod PH20 SC for pediatric patients using PK and PD results from this study.
- Phase 3: a large, late-stage study
- Time commitment: about 14 weeks
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 17
You may be able to join if
- The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written...
- The participant is aged 2 to \<18 years at the time of informed consent/assent
- The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for...
- The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable...
- The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood...
You likely can't join if
- Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant during their participation in the...
- Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication
- Has a documented lack of clinical response to plasma exchange (PLEX)
- Received a live or live-attenuated vaccine within \<4 weeks before screening
- Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study
- Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised...
See the full eligibility criteria
- The participant (and/or their legally authorized representative) understands the requirements of the study and is capable of providing written informed consent/assent and complying with protocol requirements
- The participant is aged 2 to \<18 years at the time of informed consent/assent
- The participant has been diagnosed with generalised Myasthenia Gravis that is supported by a physical examination and confirmed seropositivity for anti-acetylcholine receptor antibodies
- The participant has had an unsatisfactory response to immunosuppressants, corticosteroids, or acetylcholinesterase inhibitors but is on stable concomitant MG therapy. If receiving corticosteroids and/or...
- The participant agrees to use birth control consistent with local regulations and people of child-bearing potential must have a negative blood pregnancy test at screening and a negative urine pregnancy test before...
- Is a female adolescent of child-bearing potential who is pregnant and/or lactating or intends to become pregnant during their participation in the study
- Has worsening muscle weakness secondary to a concurrent infection or as a result of a medication
- Has a documented lack of clinical response to plasma exchange (PLEX)
- Received a live or live-attenuated vaccine within \<4 weeks before screening
- Received a thymectomy within 3 months before screening or is planning to get a thymectomy during their participation in the study
- Has a known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of generalised Myasthenia Gravis or puts the participant at undue risk
- History of malignancy, cancer, unless considered cured by adequate treatment with no evidence of recurrence for ≥3 years. Adequately treated participants with the following cancers can be included at any time: Basal...
- Clinically significant active infection that is not sufficiently resolved in the investigator's opinion or positive serum test at screening for active infection with any of the following: Hepatitis B virus (HBV)...
- Has a positive PCR test for SARS-CoV-2 at screening
- Has/had a clinically significant disease, had recent major surgery (within 3 months of screening) or intends to have major surgery during the study, or has/had any other medical condition that, in the investigator's...
- Has received a different study drug in another clinical study within \<12 before screening
- Is currently participating in another treatment clinical study
- Has previously participated in an efgartigimod clinical study and received at least one dose of study drug
- Has a known hypersensitivity to study drug or any of its excipients
- Has a history of or current episode of alcohol, drug, or medication abuse as assessed by the investigator
- Use of some medications before screening (more information is found in the protocol) The complete list of exclusion criteria can be found in the protocol.
The study team makes the final eligibility decision.
Where it's taking place
- Charlotte, North Carolina, United States
- Denton, Texas, United States
- Charlottesville, Virginia, United States
- Ghent, Belgium
- Ottawa, Canada
- Brno, Czechia
- Ostrava, Czechia
- Marseille, France
- Essen, Germany
- Jerusalem, Israel
- Petah Tikvah, Israel
- Tel Aviv, Israel
- Genova, Italy
- Leiden, Netherlands
- Gdansk, Poland
- Katowice, Poland
- Warsaw, Poland
- Valencia, Spain
- Lausanne, Switzerland
- Oxford, United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 14 weeks per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Charlotte, North Carolina, United States; Denton, Texas, United States; Charlottesville, Virginia, United States; Ghent, Belgium; Ottawa, Canada; Brno, Czechia and 14 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.